INTRODUCTION: Although prevention remains the cornerstone for the management of thalassemia, hematopoietic stem cell transplantation (HSCT) is the only curative approach.
OBJECTIVE: Our goal was to assess our experience with HSCTs for the treatment of patients with thalassemia.
METHODS: From 1994 to 2006, 96 HSCTs have been performed in 84 thalassemic children from Greece, 3 with sickle cell/thalassemia and 1 with sickle cell disease. According to Pesaro classification, of these 84 children, 20 were in class I, 35 were in class II, and 29 were in class III. Donors were 84 histocompatible siblings and 4 unrelated volunteers. The graft was of bone marrow in 85, cord blood in 3, bone marrow and cord blood in 4, and peripheral blood stem cells in 4. The conditioning regimen consisted of busulfan, cyclophosphamide, and antilymphocyte globulin.
RESULTS: All except 1 patient received engraftments. Ten patients rejected the graft. Eight received another transplant from the same donor, 7 of which were successful. Four patients died; causes of death were graft-versus-host disease (GVHD) (2), disseminated toxoplasmosis (1), and brain hemorrhage (1). At a median follow-up time of 6.5 years, 84 of 88 children survived, 81 were cured and free from transfusions, and 3 remained transfusion-dependent. Severe acute GVHD developed in 18 children, and chronic GVHD developed in 8 patients. The overall survival rate, event-free survival rate, rejection rate, and transplant-related mortality rate were 95%, 94%, 11%, and 5%, respectively. Event-free survival was 100% for class I, 95% for class II, and 87% for class III. Eleven children had mixed chimera (residual recipient hematopoiesis) with normal levels of hemoglobin.
CONCLUSIONS: HSCT is a highly effective treatment for thalassemic patients who have a fully matched donor (related or unrelated). Younger age at transplant secures excellent results with reduced morbidity and mortality rates.
Submitted by Ioulia Peristeri
- Copyright © 2008 by the American Academy of Pediatrics